ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.
ProQR Announces Second Quarter 2026 Operating and Financial Results
ProQR reported Q2 2026 revenue of €8.8 million, more than doubling the prior year, while narrowing its net loss to €8.7 million, or €0.08 per share.
The company achieved the first clinical validation of its Axiomer RNA editing platform with AX-0810 Phase 1 data showing an 8-fold increase in target engagement and a favorable safety profile.
ProQR strengthened its balance sheet with $59.2 million in financing, ending the quarter with €117.1 million in cash to fund operations into mid-2028 and support multiple upcoming clinical milestones.